News

Deal Struck to Distribute Ruconest in Middle East, North Africa

Pharming Group has signed an exclusive license agreement with NewBridge Pharmaceuticals to distribute and commercialize Ruconest (conestat alfa), a treatment for acute attacks of hereditary angioedema (HAE), in the Middle East and North Africa. Under the agreement terms, NewBridge in collaboration with Pharming, will provide access…

Early Phase 1 Data Support PHA121 as Oral HAE Prophylactic

Multiple doses of PHA121 (PHA-022121), an investigational oral therapy for hereditary angioedema (HAE), were well-tolerated and rapidly reached therapeutic levels in healthy volunteers, according to data from a Phase 1 clinical trial. The therapy’s pharmacological profile “suggests that the therapeutic effect of PHA121 can be achieved as early…

Nominations Open for 2022 Eurordis Black Pearl Awards

Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work to make a difference for the global rare disease community. The deadline for nominations is Sept. 10…

Interim Data Support Long-term Safety of Orladeyo for HAE Attacks

Treatment with Orladeyo (berotralstat) was generally safe and well-tolerated in people with hereditary angioedema (HAE), according to recently released trial data. The findings from an interim analysis of the open-label APeX-S trial also supported Orladeyo’s ability to reduce the frequency of HAE attacks, ease symptom burden, and improve…

Companies Join to Advance OTL-105, Potential Gene Therapy for HAE

Orchard Therapeutics and the Pharming Group are teaming up to research, develop, manufacture, and — possibly — commercialize a gene therapy for hereditary angioedema (HAE), called OTL-105. “This promising work toward treatment with the potential for durable long-term clinical benefit is encouraging and signifies an ongoing commitment to…

EveryLife Introduces First of Kind ‘Roadmap’ to ICD Codes

To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…

Oral On-demand KVD900 Rapidly Reduces HAE Attack Severity

KVD900, an oral experimental on-demand treatment for swelling episodes caused by hereditary angioedema (HAE), safely and significantly reduced the severity of attacks and shorted the time to improvement, which was maintained for 24 hours, according to Phase 2 trial data.  “Our goal is to provide the best outcome…